A central goal to drug discovery is the identification of a pathway associated with a trait, such as a human disease. Once a pathway has been identified, a number of approaches can be used to influence the trait, including the design of inhibitors of components (e.g., genes) of the pathway. In this way, a compound that ameliorates the effects of the trait can be developed.
The development of a compound or other entity that ameliorates the effects of a trait requires two components. First, the gene or pathway that affects a trait must be elucidated. Second, the activity of the gene or the activity of key genes in the pathway must be assayed in the presence of compounds or other entities in order to identify a compound or other entity that effectively alters the activity of the gene or affects (down regulates, up regulates) the pathway.